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Keywords = pediatric central nervous system tumors

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19 pages, 950 KiB  
Review
A Narrative Review of Theranostics in Neuro-Oncology: Advancing Brain Tumor Diagnosis and Treatment Through Nuclear Medicine and Artificial Intelligence
by Rafail C. Christodoulou, Platon S. Papageorgiou, Rafael Pitsillos, Amanda Woodward, Sokratis G. Papageorgiou, Elena E. Solomou and Michalis F. Georgiou
Int. J. Mol. Sci. 2025, 26(15), 7396; https://doi.org/10.3390/ijms26157396 - 31 Jul 2025
Viewed by 782
Abstract
This narrative review explores the integration of theranostics and artificial intelligence (AI) in neuro-oncology, addressing the urgent need for improved diagnostic and treatment strategies for brain tumors, including gliomas, meningiomas, and pediatric central nervous system neoplasms. A comprehensive literature search was conducted through [...] Read more.
This narrative review explores the integration of theranostics and artificial intelligence (AI) in neuro-oncology, addressing the urgent need for improved diagnostic and treatment strategies for brain tumors, including gliomas, meningiomas, and pediatric central nervous system neoplasms. A comprehensive literature search was conducted through PubMed, Scopus, and Embase for articles published between January 2020 and May 2025, focusing on recent clinical and preclinical advancements in personalized neuro-oncology. The review synthesizes evidence on novel theranostic agents—such as Lu-177-based radiopharmaceuticals, CXCR4-targeted PET tracers, and multifunctional nanoparticles—and highlights the role of AI in enhancing tumor detection, segmentation, and treatment planning through advanced imaging analysis, radiogenomics, and predictive modeling. Key findings include the emergence of nanotheranostics for targeted drug delivery and real-time monitoring, the application of AI-driven algorithms for improved image interpretation and therapy guidance, and the identification of current limitations such as data standardization, regulatory challenges, and limited multicenter validation. The review concludes that the convergence of AI and theranostic technologies holds significant promise for advancing precision medicine in neuro-oncology, but emphasizes the need for collaborative, multidisciplinary research to overcome existing barriers and enable widespread clinical adoption. Full article
(This article belongs to the Special Issue Biomarker Discovery and Validation for Precision Oncology)
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16 pages, 298 KiB  
Review
Small-Molecule Drugs in Pediatric Neuro-Oncology
by Stephanie Vairy and George Michaiel
Curr. Oncol. 2025, 32(8), 417; https://doi.org/10.3390/curroncol32080417 - 25 Jul 2025
Viewed by 252
Abstract
Advances in molecular diagnostics have enabled precision medicine approaches in pediatric neuro-oncology, with small-molecule drugs emerging as promising therapeutic candidates targeting specific genetic and epigenetic alterations in central nervous system (CNS) tumors. This review provides a focused overview of several small-molecule agents under [...] Read more.
Advances in molecular diagnostics have enabled precision medicine approaches in pediatric neuro-oncology, with small-molecule drugs emerging as promising therapeutic candidates targeting specific genetic and epigenetic alterations in central nervous system (CNS) tumors. This review provides a focused overview of several small-molecule agents under investigation or in early clinical use, including ONC201, tazemetostat, vorasidenib, CDK inhibitors, selinexor, and aurora kinase A inhibitors, among others. Highlighted are their mechanisms of action, pharmacokinetic properties, early efficacy data, and tolerability in pediatric populations. Despite encouraging preclinical and early-phase results, most agents face limitations due to study heterogeneity, lack of large-scale pediatric randomized trials, and challenges in drug delivery to the CNS. The review underscores the critical need for robust prospective clinical trials for the integration of these therapies into pediatric neuro-oncology care. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
26 pages, 359 KiB  
Review
Old Tools in a New Era: The Continued Relevance of Chemotherapy in Pediatric Neuro-Oncology
by Kathleen Felton, Lucie Lafay-Cousin and Sylvia Cheng
Curr. Oncol. 2025, 32(7), 410; https://doi.org/10.3390/curroncol32070410 - 20 Jul 2025
Viewed by 406
Abstract
Conventional chemotherapy continues to form the backbone of treatment for many pediatric central nervous system (CNS) tumors. Advances have been made especially in the molecular underpinning of certain pediatric CNS tumors, allowing for advancement and consideration in incorporating this molecular information in molecular [...] Read more.
Conventional chemotherapy continues to form the backbone of treatment for many pediatric central nervous system (CNS) tumors. Advances have been made especially in the molecular underpinning of certain pediatric CNS tumors, allowing for advancement and consideration in incorporating this molecular information in molecular targeted therapy or appropriate de-escalation or escalation of therapy. In very young children with embryonal CNS tumors, intensive high-dose chemotherapy approaches have been used with varied increased survival in medulloblastoma, atypical teratoid rhabdoid tumor (ATRT), and rare embryonal subtypes, but there are certain molecular risk groups that require new therapies, such as the ATRT MYC subtype. Some CNS tumors remain resistant or refractory to conventional chemotherapy, especially in relapsed disease. Strategies to explore combination therapies with chemotherapy, novel agents, and novel approaches are needed to improve survival in this population in the future. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
12 pages, 1397 KiB  
Review
The Current Landscape of Molecular Pathology for the Diagnosis and Treatment of Atypical Teratoid Rhabdoid Tumor
by Ashley Childress, Alayna Koch, Emma Vallee, Alyssa Steller and Scott Raskin
J. Mol. Pathol. 2025, 6(2), 13; https://doi.org/10.3390/jmp6020013 - 14 Jun 2025
Viewed by 630
Abstract
Atypical teratoid rhabdoid tumor (ATRT) is a rare, aggressive pediatric central nervous system (CNS) tumor that predominantly affects children under the age of 3. It is defined by the inactivation of the SMARCB1 gene, leading to the loss of INI1, a protein essential [...] Read more.
Atypical teratoid rhabdoid tumor (ATRT) is a rare, aggressive pediatric central nervous system (CNS) tumor that predominantly affects children under the age of 3. It is defined by the inactivation of the SMARCB1 gene, leading to the loss of INI1, a protein essential for cell lineage determination and cell differentiation. Current standard of care treatment requires aggressive multimodal therapy with maximal safe resection, high-dose chemotherapy with autologous stem cell rescue, and radiation, yet overall survival remains < 50%. These intensive regimens have improved overall survival but are associated with significant morbidity and long-term effects. Molecular profiling has significantly advanced the understanding of ATRTs, revealing four molecular subgroups, ATRT-TYR, ATRT-MYC, ATRT-SHH, and ATRT-SMARCA4, each with distinct clinical presentations, oncogenic pathways, and prognoses. Molecular characterization enables better prognostic stratification, guiding treatment decisions and allowing for more personalized therapeutic approaches. Targeted therapies based on these molecular insights remain experimental, and continued exploration of molecular mechanisms and how they differ amongst subgroups is pivotal for the development of less toxic, more effective targeted treatments. Full article
(This article belongs to the Collection Feature Papers in Journal of Molecular Pathology)
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23 pages, 1894 KiB  
Review
From Seeing to Healing: The Clinical Potential of Radiotracers in Pediatric Neuro-Oncology
by Bojana Bogdanović and Christopher Montemagno
Cancers 2025, 17(12), 1905; https://doi.org/10.3390/cancers17121905 - 7 Jun 2025
Viewed by 783
Abstract
Pediatric central nervous system (CNS) tumors, including gliomas, medulloblastomas, and diffuse midline gliomas (previously diffuse intrinsic pontine gliomas), remain a major clinical challenge due to their complex biology, limited treatment effectiveness, and generally poor prognosis. Standard treatments are often aggressive and associated with [...] Read more.
Pediatric central nervous system (CNS) tumors, including gliomas, medulloblastomas, and diffuse midline gliomas (previously diffuse intrinsic pontine gliomas), remain a major clinical challenge due to their complex biology, limited treatment effectiveness, and generally poor prognosis. Standard treatments are often aggressive and associated with substantial toxicity, particularly in advanced stages. This review highlights recent developments in radiopharmaceuticals for molecular imaging and targeted radiotherapy. A comprehensive literature analysis was conducted, focusing on radiotracers with clinical relevance in pediatric neuro-oncology, including metabolic, peptide receptor-based, and antibody-based agents. Radiopharmaceuticals such as 18F-FLT, 64CuCl2, and 1-L-18F-FETrp have improved the ability to monitor tumor biology, proliferation, and treatment response, aiding in diagnosis at an early stage, assessment of tumor behavior, and detection of recurrence or progression. Additionally, peptide receptor-based radiotracers, such as 68Ga-DOTATATE and 177Lu-DOTATATE, are already used for both diagnostic purposes and targeted radiotherapy, particularly in neuroblastomas and gliomas. Antibody-based radiotracers like 131I-omburtamab, targeting B7-H3, are emerging as promising tools for addressing difficult-to-treat tumors such as diffuse midline glioma. Collectively, these advances provide new hope for children afflicted by these devastating malignancies, offering promising solutions for more specific and precise diagnosis and, additionally, for more effective, personalized, and less toxic tumor therapies. Full article
(This article belongs to the Special Issue Pediatric Brain Tumors: Symptoms, Diagnosis and Treatments)
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14 pages, 606 KiB  
Review
Approaches to Reduce Toxicity in Pediatric Brain Tumors
by Hallie Coltin, Christina Coleman and Chantel Cacciotti
Curr. Oncol. 2025, 32(5), 281; https://doi.org/10.3390/curroncol32050281 - 15 May 2025
Viewed by 661
Abstract
Pediatric central nervous system (CNS) tumor survivors are highly susceptible to long-term toxicity due to tumor location and also the treatment received. Advancements in treatment techniques, risk-adapted approaches to therapy with adjustments to treatment regimens—including de-escalation when feasible—along with the addition of supportive [...] Read more.
Pediatric central nervous system (CNS) tumor survivors are highly susceptible to long-term toxicity due to tumor location and also the treatment received. Advancements in treatment techniques, risk-adapted approaches to therapy with adjustments to treatment regimens—including de-escalation when feasible—along with the addition of supportive therapy and surveillance in these survivors, serve to minimize and manage late effects of therapy. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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45 pages, 15819 KiB  
Review
The Molecular Basis of Pediatric Brain Tumors: A Review with Clinical Implications
by Elias Antoniades, Nikolaos Keffes, Stamatia Vorri, Vassilios Tsitouras, Nikolaos Gkantsinikoudis, Parmenion Tsitsopoulos and John Magras
Cancers 2025, 17(9), 1566; https://doi.org/10.3390/cancers17091566 - 4 May 2025
Viewed by 2061
Abstract
Central nervous system (CNS) tumors are the most common solid malignancy in the pediatric population. These lesions are the result of the aberrant cell signaling step proteins, which normally regulate cell proliferation. Mitogen-activated protein kinase (MAPK) pathways and tyrosine kinase receptors are involved [...] Read more.
Central nervous system (CNS) tumors are the most common solid malignancy in the pediatric population. These lesions are the result of the aberrant cell signaling step proteins, which normally regulate cell proliferation. Mitogen-activated protein kinase (MAPK) pathways and tyrosine kinase receptors are involved in tumorigenesis of low-grade gliomas. High-grade gliomas may carry similar mutations, but loss of epigenetic control is the dominant molecular event; it can occur either due to histone mutations or inappropriate binding or unbinding of DNA on histones. Therefore, despite the absence of genetic alteration in the classic oncogenes or tumor suppressor genes, uncontrolled transcription results in tumorigenesis. Isocitric dehydrogenase (IDH) mutations do not predominate compared to their adult counterpart. Embryonic tumors include medulloblastomas, which bear mutations of transcription-regulating pathways, such as wingless-related integration sites or sonic hedgehog pathways. They may also relate to high expression of Myc family genes. Atypical teratoid rhabdoid tumors harbor alterations of molecules that contribute to ATP hydrolysis of chromatin. Embryonic tumors with multilayered rosettes are associated with microRNA mutations and impaired translation. Ependymomas exhibit great variability. As far as supratentorial lesions are concerned, the major events are mutations either of NFkB or Hippo pathways. Posterior fossa tumors are further divided into two types with different prognoses. Type A group is associated with mutations of DNA damage repair molecules. Lastly, germ cell tumors are a heterogeneous group. Among them, germinomas manifest KIT receptor mutations, a subgroup of the tyrosine kinase receptor family. Full article
(This article belongs to the Special Issue New Advances in the Treatment of Pediatric Solid Tumors)
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14 pages, 2479 KiB  
Article
Primary Central Nervous System Tumors in Adolescents: A Population-Based Study on Epidemiology and Clinical Pathways in a Challenging Age Group
by Lucia De Martino, Patrizia Piga, Marcella Sessa, Camilla Calì, Camilla Russo, Stefania Picariello, Nicola Onorini, Pietro Spennato, Lucia Quaglietta, Maria Vittoria Donofrio, Giuseppe Cinalli, Francesco Vetrano and Fabio Savoia
Curr. Oncol. 2025, 32(4), 222; https://doi.org/10.3390/curroncol32040222 - 10 Apr 2025
Viewed by 601
Abstract
Background: Oncological care of adolescent patients is often inconsistent, as they frequently fall between pediatric and adult services. The Childhood Cancer Registry of Campania (CCRC) is the Italian largest population-based registry specializing in children 0–19 years old, with a target population of approximately [...] Read more.
Background: Oncological care of adolescent patients is often inconsistent, as they frequently fall between pediatric and adult services. The Childhood Cancer Registry of Campania (CCRC) is the Italian largest population-based registry specializing in children 0–19 years old, with a target population of approximately 1.1 million inhabitants. Material and Methods: This report presents epidemiological indicators and clinical pathways on primary brain tumors in adolescents (15–19 years) from the Campania region. Results: Over the study period (2008–2020), the cohort included 219 adolescents with newly diagnosed central nervous system (CNS) tumors with an annual average incidence rate (IR) of 48.9 cases per million/year. The 5-year observed survival rate after diagnosis of CNS tumor was 84.8%. Overall, the most common tumor site was the pituitary gland and craniopharyngeal duct, representing 22.4% of all tumors. The most frequently occurring malignant primary CNS tumor was germinoma, while the most common non-malignant tumor was pituitary adenoma. Most patients were referred to adult services and nearly half migrated outside the region to receive cancer care. Conclusions: Challenges in the care of adolescent oncology patients include limited access to specialized care, difficulties in transitioning from pediatric to adult institutions, distinct tumor biology, and the underrepresentation of adolescents in clinical trials. The care of adolescents with CNS tumors is fragmented across institutions and significant variations in practice exist between adult and pediatric practitioners. Full article
(This article belongs to the Special Issue Quality of Life and Follow-Up Care Among AYA Cancer Survivors)
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14 pages, 2481 KiB  
Article
Evaluating the Potential of PSMA Targeting in CNS Tumors: Insights from Large-Scale Transcriptome Profiling
by Adam Kraya, Komal Rathi, Run Jin, Varun Kesherwani, Adam C. Resnick, Phillip B. Storm and Ali Nabavizadeh
Cancers 2025, 17(7), 1239; https://doi.org/10.3390/cancers17071239 - 6 Apr 2025
Viewed by 869
Abstract
Background/Objectives: Prostate-specific membrane antigen (PSMA) is a well-established target in prostate cancer therapy that has shown potential as a theranostic target across non-central nervous system (CNS) and CNS tumor types. We aimed to investigate the pan-tissue expression pattern of the PSMA-encoding gene FOLH1 [...] Read more.
Background/Objectives: Prostate-specific membrane antigen (PSMA) is a well-established target in prostate cancer therapy that has shown potential as a theranostic target across non-central nervous system (CNS) and CNS tumor types. We aimed to investigate the pan-tissue expression pattern of the PSMA-encoding gene FOLH1 to assess whether transcriptome profiling can inform tumor diagnostic and theranostic probes. Methods: We assessed FOLH1 expression from the Open Pediatric Cancer Project (OpenPedCan, n = 2132 specimens), the Cancer Genome Atlas (TCGA, n = 10,411 specimens), and the Genotype Tissue Expression Project (GTEx, n = 17,382 specimens) in relation to published reports of PSMA radionuclide uptake in various tumors. Results: When comparing FOLH1 expression across tumor versus normal tissues, we found that non-CNS tumors exhibiting elevated expression of at least two-fold (FDR < 0.05) were reported to have significant PSMA radionuclide uptake in contrast to tumors with less than a two-fold elevation or with lower expression of FOLH1 relative to normal tissues. Notably, CNS tumors universally exhibited lower expression of FOLH1 relative to normal brain tissue, but we observed considerable variation in the expression of blood–tumor barrier (BTB) components associated with reports of BTB integrity and uptake of PSMA radiotracers. Conclusions: Large-scale transcriptomics data may help guide the application of PSMA-based radionuclide therapies in non-CNS tumors, but care should be taken to account for BTB effects in CNS tumors when assessing the potential for radionuclide success. This study demonstrates that FOLH1 showed a lack of tumor-specific expression for both adult and pediatric CNS tumors when compared to normal brain tissue, suggesting that PSMA is not a desirable target in brain tumors. Full article
(This article belongs to the Special Issue Molecular Pathology of Brain Tumors)
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15 pages, 1370 KiB  
Review
Can Focused Ultrasound Overcome the Failure of Chemotherapy in Treating Pediatric Diffuse Intrinsic Pontine Glioma Due to a Blood–Brain Barrier Obstacle?
by Silvana Filieri, Morena Miciaccia, Domenico Armenise, Olga Maria Baldelli, Anselma Liturri, Savina Ferorelli, Anna Maria Sardanelli, Maria Grazia Perrone and Antonio Scilimati
Pharmaceuticals 2025, 18(4), 525; https://doi.org/10.3390/ph18040525 - 3 Apr 2025
Cited by 1 | Viewed by 943
Abstract
Background: The blood–brain barrier (BBB) plays an important role in regulating homeostasis of the central nervous system (CNS), and it is an obstacle for molecules with a molecular weight higher than 500 Da seeking to reach it, making many drugs ineffective simply [...] Read more.
Background: The blood–brain barrier (BBB) plays an important role in regulating homeostasis of the central nervous system (CNS), and it is an obstacle for molecules with a molecular weight higher than 500 Da seeking to reach it, making many drugs ineffective simply because they cannot be delivered to where they are needed. As a result, crossing the BBB remains the rate-limiting factor in brain drug delivery during the treatment of brain diseases, specifically tumors such as diffuse intrinsic pontine glioma (DIPG), a highly aggressive pediatric tumor with onset in the pons Varolii, the middle portion of the three contiguous parts of the brainstem, located above the medulla and below the midbrain. Methods: Currently, radiotherapy (RT) relieves DIPG symptoms but chemotherapy drugs do not lead to significant results as they do not easily cross the BBB. Focused ultrasound (FUS) and microbubbles (MBs) can temporarily open the BBB, facilitating radiotherapy and the entry of drugs into the CNS. A patient-derived xenograft DIPG model exposed to high-intensity focalized ultrasound (HIFU) or low-intensity focalized ultrasound (LIFU) combined with MBs was treated with doxorubicin, panobinostat, olaparib, ONC201 (Dordaviprone®) and anti-PD1. Panobinostat has also been used in children with diffuse midline glioma, a broad class of brain tumors to which DIPG belongs. Results: Preliminary studies were performed using FUS to temporarily open the BBB and allow a milder use of radiotherapy and facilitate the passage of drugs through the BBB. The data collected show that after opening the BBB with FUS and MBs, drug delivery to the CNS significantly improved. Conclusions: FUS associated with MBs appears safe and feasible and represents a new strategy to increase the uptake of drugs in the CNS and therefore enhance their effectiveness. This review reports pre-clinical and clinical studies performed to demonstrate the usefulness of FUS in patients with DIPG treated with some chemotherapy. The papers reviewed were published in PubMed until the end of 2024 and were found using a combination of the following keywords: diffuse intrinsic pontine glioma (DIPG), DIPG H3K27-altered, blood–brain barrier and BBB, focused ultrasound (FUS) and radiotherapy (RT). Full article
(This article belongs to the Section Radiopharmaceutical Sciences)
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14 pages, 488 KiB  
Review
Tumor-Treating Fields and Related Treatments in the Management of Pediatric Brain Tumors
by Julien Rousseau, Sarah Lapointe and David Roberge
Curr. Oncol. 2025, 32(4), 185; https://doi.org/10.3390/curroncol32040185 - 21 Mar 2025
Viewed by 1633
Abstract
Pediatric primary brain tumors pose significant therapeutic challenges due to their aggressive nature and the critical environment of the developing brain. Traditional modalities like surgery, chemotherapy, and radiotherapy often achieve limited success in high-grade gliomas and embryonal tumors. Tumor-treating fields (TTfields), a non-invasive [...] Read more.
Pediatric primary brain tumors pose significant therapeutic challenges due to their aggressive nature and the critical environment of the developing brain. Traditional modalities like surgery, chemotherapy, and radiotherapy often achieve limited success in high-grade gliomas and embryonal tumors. Tumor-treating fields (TTfields), a non-invasive therapy delivering alternating electric fields, has emerged as a promising approach to disrupt tumor cell division through mechanisms such as mitotic disruption, DNA damage, and tumor microenvironment modulation. TTfields are thought to selectively target dividing tumor cells while sparing healthy, non-dividing cells. While TTfields therapy is FDA-approved for the management of glioblastoma and other cancers, its application in pediatric brain tumors remains under investigation. Preclinical studies reveal its potential in medulloblastoma and ependymoma models, while observational data suggest its safety and feasibility in children. Current research focuses on optimizing TTfields’ efficacy through advanced technologies, including high-intensity arrays, skull remodeling, and integration with immunotherapies such as immune checkpoint inhibitors. Innovative device-based therapies like magnetic field-based technologies further expand the treatment possibilities. As clinical trials progress, TTfields and related modalities offer hope for addressing unmet needs in pediatric neuro-oncology, especially for tumors in challenging locations. Future directions include biomarker identification, tailored protocols, and novel therapeutic combinations to enhance outcomes in pediatric brain tumor management. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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28 pages, 1398 KiB  
Review
Pediatric CNS Radiation Oncology: Recent Developments and Novel Techniques
by Justin Oh, Samir Patel, Mary-Pat Schlosser, Andrew J. Arifin, Carol Oliveira, Anne-Marie Charpentier and Derek S. Tsang
Curr. Oncol. 2025, 32(3), 180; https://doi.org/10.3390/curroncol32030180 - 20 Mar 2025
Cited by 1 | Viewed by 1364
Abstract
Radiation therapy (RT) is a cornerstone in the management of pediatric central nervous system (CNS) tumors. Recent advancements in RT delivery and techniques aim to enhance therapeutic effectiveness while minimizing both acute and long-term complications associated with pediatric brain RT. This paper highlights [...] Read more.
Radiation therapy (RT) is a cornerstone in the management of pediatric central nervous system (CNS) tumors. Recent advancements in RT delivery and techniques aim to enhance therapeutic effectiveness while minimizing both acute and long-term complications associated with pediatric brain RT. This paper highlights innovative developments in the field, including the clinical indications, benefits, and challenges of proton therapy and stereotactic radiotherapy. The ongoing refinement of risk-adapted RT volumes is highlighted, with examples of newly proposed germinoma RT volumes and hippocampal-sparing RT. Additionally, emerging experimental approaches, including FLASH therapy and theranostics, are also discussed as promising future directions. Further prospective, multi-institutional collaborative studies are essential to validate and expand upon the benefits outlined in this review. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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11 pages, 645 KiB  
Article
Characterizing the Natural History of Pediatric Brain Tumors Presenting with Metastasis
by Victor M. Lu and Toba N. Niazi
Cancers 2025, 17(5), 775; https://doi.org/10.3390/cancers17050775 - 24 Feb 2025
Viewed by 689
Abstract
Background: The natural history of pediatric patients with metastasis of primary brain tumors within and outside the central nervous system is poorly understood, as too are possible clinical correlates with outcome. Correspondingly, the aim of this study was to interrogate a national database [...] Read more.
Background: The natural history of pediatric patients with metastasis of primary brain tumors within and outside the central nervous system is poorly understood, as too are possible clinical correlates with outcome. Correspondingly, the aim of this study was to interrogate a national database to characterize this diagnosis and its clinical course in pediatric patients. Methods: The U.S. National Cancer Database (NCDB) was interrogated between the years 2005–2016 for all patients aged 18 years and younger with a primary brain tumor diagnosis, as well as evidence of disease metastasis at initial diagnosis. Data were summarized and overall survival (OS) was modeled using Kaplan–Meier and Cox regression analyses. Results: Out of a total of 8615 pediatric brain tumor patients, 356 (4%) had evidence of metastasis at initial diagnosis. Compared to patients without metastasis, patients with metastasis were statistically younger, more often male, and less likely to have private health insurance (all p < 0.050). With respect to clinical characteristics, the primary tumors of patients with metastasis were statistically more likely to be located in the cerebellum; be of higher histologic grading, with a higher proportion of medulloblastoma diagnoses and lower proportion of malignant glioma and pilocytic astrocytoma diagnoses; and were more likely to be treated by subtotal surgical resection, chemotherapy and radiation therapy (all p < 0.050) when compared to patients without metastasis. Five-year OS for those with metastasis was significantly lower than those without (48% vs. 75%, p < 0.001), with the median overall survival for patients with metastasis being 53 months (95% CI 29–86). Multivariate analysis indicated that a shorter OS was independently associated with the primary diagnoses of malignant glioma (HR 27.7, p = 0.020) and Atypical Teratoid/Rhabdoid Tumor (ATRT, HR 41.1, p = 0.041) and with WHO grades 3 (HR 20.1, p = 0.012) and 4 (HR 11.5, p < 0.001). Longer OS was significantly and independently associated with surgery (HR 0.49, p < 0.001), chemotherapy (HR 0.53, p = 0.041), and radiation therapy (HR 0.57, p = 0.026). Conclusions: Although uncommon, pediatric brain tumors with evidence of metastasis at initial diagnosis will present with a distinct socioeconomic and clinical profile compared to patients without metastasis. Multiple predictors are independently associated with overall prognosis, and understanding these features should be validated in prospective efforts to identify vulnerable patients earlier in order to maximize the impact of treatment. Full article
(This article belongs to the Special Issue Brain Metastases: From Mechanisms to Treatment)
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23 pages, 3717 KiB  
Article
Epidemiology and Outcome of Primary Central Nervous System Tumors Treated at King Hussein Cancer Center
by Maysa Al-Hussaini, Abdallah Al-Ani, Justin Z. Amarin, Sarah Al Sharie, Mouness Obeidat, Awni Musharbash, Amer A. Al Shurbaji, Ahmad Kh. Ibrahimi, Abdellatif Al-Mousa, Nasim Sarhan, Nisreen Amayiri, Rula Amarin, Tala Alawabdeh, Qasem Alzoubi, Dima Abu Laban, Bayan Maraqa, Khaled Jamal and Asem Mansour
Cancers 2025, 17(4), 590; https://doi.org/10.3390/cancers17040590 - 9 Feb 2025
Viewed by 1573
Abstract
Background and Objectives: Primary central nervous system (CNS) tumors are often associated with relatively poor outcomes. Data on the epidemiology and outcome of CNS tumors in Jordan are scarce. We aim to report the epidemiology and outcome of primary CNS tumors of patients [...] Read more.
Background and Objectives: Primary central nervous system (CNS) tumors are often associated with relatively poor outcomes. Data on the epidemiology and outcome of CNS tumors in Jordan are scarce. We aim to report the epidemiology and outcome of primary CNS tumors of patients managed at a comprehensive cancer care center in Jordan. Methods: We performed a retrospective chart review of all Jordanian patients with a primary CNS tumor diagnosis who were managed at the center between July 2003 and June 2019. We included all entities described in the 2021 CNS WHO classification system, in addition to pituitary neuroendocrine tumors (PitNETs). We used the Kaplan–Meier method to estimate the 1-year, 2-year, and 5-year overall survival (OS) rates for each entity. Results and Findings: We included 2094 cases. The numbers of pediatrics and adults were 652 (31.1%) and 1442 (68.9%), respectively. The three most common groups of tumors were “gliomas, glioneuronal tumors, and neuronal tumors” (n = 1200 [57.30%]), followed by meningiomas (n = 261 [12.5%]), embryonal tumors (n = 234 [11.2%]). The three most common tumor families were adult-type diffuse gliomas (n = 709 [33.8%]), medulloblastoma (n = 199 [9.5%]), and circumscribed astrocytic gliomas (n = 183 [8.7%]). The median survival for the entire cohort was 97 months (95CI; 81–112). Survival was significantly worse for males and adults compared to their respective counterparts. Among the most common tumor group, “gliomas, glioneuronal tumors, and neuronal tumors”, OS rates for adult-type diffuse gliomas were significantly lower than all other types. Overall, adult gliomas with IDH-mutations had a survival advantage over wildtype cases (IDH-mutant 1-year OS, 89% [82–97%] vs. IDH-wildtype 1-year OS, 60% [52–70%]; p < 0.001). Conclusions: We present a detailed analysis of the primary CNS tumors diagnosed in the largest cancer center in Jordan between 2003 and 2019. We compared the epidemiology and overall survival of these patients to worldwide estimates and found the epidemiology and outcome of these tumors comparable to worldwide trends. Full article
(This article belongs to the Section Cancer Epidemiology and Prevention)
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34 pages, 1418 KiB  
Review
Advances in the Repurposing and Blood–Brain Barrier Penetrance of Drugs in Pediatric Brain Tumors
by Julian S. Rechberger, Stephanie A. Toll, Subhasree Biswas, Hyo Bin You, William D. Chow, Nicholas Kendall, Pournima Navalkele and Soumen Khatua
Cancers 2025, 17(3), 439; https://doi.org/10.3390/cancers17030439 - 27 Jan 2025
Cited by 1 | Viewed by 3061
Abstract
Central nervous system (CNS) tumors are the leading cause of cancer-related mortality in children, with prognosis remaining dismal for some of these malignancies. Though the past two decades have seen advancements in surgery, radiation, and targeted therapy, major unresolved hurdles continue to undermine [...] Read more.
Central nervous system (CNS) tumors are the leading cause of cancer-related mortality in children, with prognosis remaining dismal for some of these malignancies. Though the past two decades have seen advancements in surgery, radiation, and targeted therapy, major unresolved hurdles continue to undermine the therapeutic efficacy. These include challenges in suboptimal drug delivery through the blood–brain barrier (BBB), marked intra-tumoral molecular heterogeneity, and the elusive tumor microenvironment. Drug repurposing or re-tasking FDA-approved drugs with evidence of penetration into the CNS, using newer methods of intracranial drug delivery facilitating optimal drug exposure, has been an area of intense research. This could be a valuable tool, as most of these agents have already gone through the lengthy process of drug development and the evaluation of safety risks and the optimal pharmacokinetic profile. They can now be used and tested in clinics with an accelerated and different approach. Conclusions: The next-generation therapeutic strategy should prioritize repurposing oncologic and non-oncologic drugs that have been used for other indication, and have demonstrated robust preclinical activity against pediatric brain tumors. In combination with novel drug delivery techniques, these drugs could hold significant therapeutic promise in pediatric neurooncology. Full article
(This article belongs to the Special Issue Advances in Drug Repurposing to Overcome Cancers)
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